BioMarin gene therapy for haemophilia on course for EU appro...

The EMA's human medicines advisory committee has recommended that BioMarin Pharmaceutical's haemophilia A gene therapy Roctavian be approved in the EU, removing one of the last barriers to

NICE firms up NHS use of Vimizim for Morquio A syndrome

In 2015, NICE said that BioMarin's Vimizim could be used to treat patients with the ultra-rare disease Morquio A syndrome, while real-world data was collected on its efficacy.

FDA slaps clinical hold on BioMarin's PKU gene therapy

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PK

2nd Gene Therapy for Blood Disorders

Gene Therapy for Blood Disorders Digital Summit

BioMarin files achondroplasia drug after rejection for haemo...

US biotech BioMarin has hit back with a filing for a rare disease drug, after suffering what was described by analysts as a “major setback” when the FDA rejected its gene therapy for haemop

Girl loses High Court battle over NICE review of BioMarin’s ...